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Published: Mar, 2015 | Pages:
57 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2015 Summary Global Markets Direct’s, ‘Paroxysmal Nocturnal Hemoglobinuria - Pipeline Review, H1 2015’, provides an overview of the Paroxysmal Nocturnal Hemoglobinuria’s therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Paroxysmal Nocturnal Hemoglobinuria, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Paroxysmal Nocturnal Hemoglobinuria and special features on late-stage and discontinued projects. Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Paroxysmal Nocturnal Hemoglobinuria - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Paroxysmal Nocturnal Hemoglobinuria and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Paroxysmal Nocturnal Hemoglobinuria products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Paroxysmal Nocturnal Hemoglobinuria pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Paroxysmal Nocturnal Hemoglobinuria - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Paroxysmal Nocturnal Hemoglobinuria pipeline depth and focus of Indication therapeutics - Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope - Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Contents Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Paroxysmal Nocturnal Hemoglobinuria Overview 6 Therapeutics Development 7 Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Overview 7 Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Comparative Analysis 8 Paroxysmal Nocturnal Hemoglobinuria - Therapeutics under Development by Companies 9 Paroxysmal Nocturnal Hemoglobinuria - Pipeline Products Glance 10 Clinical Stage Products 10 Early Stage Products 11 Paroxysmal Nocturnal Hemoglobinuria - Products under Development by Companies 12 Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development 13 Achillion Pharmaceuticals, Inc. 13 Alnylam Pharmaceuticals, Inc. 14 Amyndas Pharmaceuticals LLC 15 Apellis Pharmaceuticals, Inc. 16 Omeros Corporation 17 Ra Pharmaceuticals, Inc. 18 Shire Plc 19 Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 20 Assessment by Monotherapy Products 20 Assessment by Target 21 Assessment by Mechanism of Action 23 Assessment by Route of Administration 25 Assessment by Molecule Type 27 Drug Profiles 29 ACH-CFDIS - Drug Profile 29 Product Description 29 Mechanism of Action 29 R&D Progress 29 ALN-CC5 - Drug Profile 30 Product Description 30 Mechanism of Action 30 R&D Progress 30 AMY-101 - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 APL-2 - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 C1 esterase inhibitor (human) - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 Coversin - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 37 Product Description 37 Mechanism of Action 37 R&D Progress 37 NM-9405 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 OMS-721 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 RA-101348 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 Paroxysmal Nocturnal Hemoglobinuria - Recent Pipeline Updates 42 Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 51 Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 52 Featured News & Press Releases 52 Dec 04, 2014: Ra Pharmaceuticals Announces Presentation of Data Supporting Clinical Development of Novel Complement C5 Inhibitor at 2014 ASH Annual Meeting 52 Mar 28, 2013: Omeros Announces Toxicology Data That Support Advancing MASP-2 Inhibitor Into Clinical Trials 53 Feb 07, 2013: Omeros Announces Toxicology Study Data From MASP-2 Inhibitor 54 Appendix 56 Methodology 56 Coverage 56 Secondary Research 56 Primary Research 56 Expert Panel Validation 56 Contact Us 56 Disclaimer 57" Table of Contents Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Paroxysmal Nocturnal Hemoglobinuria Overview 6 Therapeutics Development 7 Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Overview 7 Pipeline Products for Paroxysmal Nocturnal Hemoglobinuria - Comparative Analysis 8 Paroxysmal Nocturnal Hemoglobinuria - Therapeutics under Development by Companies 9 Paroxysmal Nocturnal Hemoglobinuria - Pipeline Products Glance 10 Clinical Stage Products 10 Early Stage Products 11 Paroxysmal Nocturnal Hemoglobinuria - Products under Development by Companies 12 Paroxysmal Nocturnal Hemoglobinuria - Companies Involved in Therapeutics Development 13 Achillion Pharmaceuticals, Inc. 13 Alnylam Pharmaceuticals, Inc. 14 Amyndas Pharmaceuticals LLC 15 Apellis Pharmaceuticals, Inc. 16 Omeros Corporation 17 Ra Pharmaceuticals, Inc. 18 Shire Plc 19 Paroxysmal Nocturnal Hemoglobinuria - Therapeutics Assessment 20 Assessment by Monotherapy Products 20 Assessment by Target 21 Assessment by Mechanism of Action 23 Assessment by Route of Administration 25 Assessment by Molecule Type 27 Drug Profiles 29 ACH-CFDIS - Drug Profile 29 Product Description 29 Mechanism of Action 29 R&D Progress 29 ALN-CC5 - Drug Profile 30 Product Description 30 Mechanism of Action 30 R&D Progress 30 AMY-101 - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 APL-2 - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 C1 esterase inhibitor (human) - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 Coversin - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 Monoclonal Antibody for Paroxysmal Nocturnal Hemoglobinuria - Drug Profile 37 Product Description 37 Mechanism of Action 37 R&D Progress 37 NM-9405 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 OMS-721 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 RA-101348 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 Paroxysmal Nocturnal Hemoglobinuria - Recent Pipeline Updates 42 Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects 51 Paroxysmal Nocturnal Hemoglobinuria - Product Development Milestones 52 Featured News & Press Releases 52 Dec 04, 2014: Ra Pharmaceuticals Announces Presentation of Data Supporting Clinical Development of Novel Complement C5 Inhibitor at 2014 ASH Annual Meeting 52 Mar 28, 2013: Omeros Announces Toxicology Data That Support Advancing MASP-2 Inhibitor Into Clinical Trials 53 Feb 07, 2013: Omeros Announces Toxicology Study Data From MASP-2 Inhibitor 54 Appendix 56 Methodology 56 Coverage 56 Secondary Research 56 Primary Research 56 Expert Panel Validation 56 Contact Us 56 Disclaimer 57
List of Tables Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria, H1 2015 7 Number of Products under Development for Paroxysmal Nocturnal Hemoglobinuria - Comparative Analysis, H1 2015 8 Number of Products under Development by Companies, H1 2015 9 Comparative Analysis by Clinical Stage Development, H1 2015 10 Comparative Analysis by Early Stage Development, H1 2015 11 Products under Development by Companies, H1 2015 12 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Achillion Pharmaceuticals, Inc., H1 2015 13 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2015 14 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Amyndas Pharmaceuticals LLC, H1 2015 15 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Apellis Pharmaceuticals, Inc., H1 2015 16 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Omeros Corporation, H1 2015 17 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Ra Pharmaceuticals, Inc., H1 2015 18 Paroxysmal Nocturnal Hemoglobinuria - Pipeline by Shire Plc, H1 2015 19 Assessment by Monotherapy Products, H1 2015 20 Number of Products by Stage and Target, H1 2015 22 Number of Products by Stage and Mechanism of Action, H1 2015 24 Number of Products by Stage and Route of Administration, H1 2015 26 Number of Products by Stage and Molecule Type, H1 2015 28 Paroxysmal Nocturnal Hemoglobinuria Therapeutics - Recent Pipeline Updates, H1 2015 42 Paroxysmal Nocturnal Hemoglobinuria - Dormant Projects, H1 2015 51
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